Trial Watch: VX-264 For Type 1 Diabetes Discontinued
Vertex Pharmaceuticals recently announced the discontinuation of its VX-264 trial, which tested whether lab-grown islet cells transplanted into a small protective device could produce insulin in people with type 1 diabetes. The VX-264 trial showed the treatment was safe, but the cells in the device didn't produce enough insulin to continue.
Despite this setback, Vertex says it remains committed to advancing device-based approaches and other strategies, such as gene editing, to protect transplanted islet cells without the need for immunosuppressive therapy.
At the same time, progress continues with Vertex’s Zimislecel (formerly VX-880), a cell-based therapy that does require immunosuppression. This treatment is aimed at individuals with type 1 diabetes who experience hypoglycemic unawareness. Encouraged by recent results, the company has accelerated its timeline for FDA submission from 2030 to 2026. With FDA fast track designation, Zimislecel could become available as early as 2027.
Trial status: Discontinued
Clinical Trials Identifier: NCT05791201
Trial Name: A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes
Diabetes Type: Type 1
Trial Sponsor: Vertex Pharmaceuticals / UHealth Diabetes Research Institute
What was the VX-264 trial researching?
While islet cell transplantation is one of the most promising potential cures for type 1 diabetes, the body’s immune system response will attack the transplanted cells, presenting a daunting challenge. The transplantation method used in the VX-264 study aimed to provide years of blood sugar control akin to a functioning pancreas – but without the need for immunosuppressants.
In the VX-264 study, the cell-derived islet cells were encapsulated with a device to protect them from the recipient’s immune system. In a surgical procedure under general anesthesia, the devices are implanted behind the muscles in the abdominal wall.
A related trial, the VX-880 study – which uses the same cell-derived beta cells, but does require the use of immunosuppressive drugs to fight off rejection – has shown promising results. Vertex recently announced that all 12 of the patients in the study began producing insulin by day 90. The three patients who had passed the year mark after treatment have continued to produce insulin.
Why is this research important?
Current islet transplantation methods require the person who receives the transplant to take lifelong immunosuppressive drugs to protect the transplanted cells. The drugs, which are taken daily, come with additional risks of infection and certain types of cancer.
In addition, using current transplant methods, nearly 60% of the cells are rejected by the body. The VX-264 study was designed to alleviate the need for anti-rejection drugs, while the encapsulation protects the cells and their ability to regulate blood sugar and deliver insulin. The novel aspect of the treatment was an encapsulation device that protects the islet cells from the body’s immune system.
The VX-264 trial was one of the most anticipated research projects in years for people with type 1 diabetes. Despite it being discontinued, Vertex is now analyzing the VX-264 trial results and will continue research on therapies with and without the need for immunosuppressive drugs.
Learn more about new treatments for type 1 diabetes here: